OrphanPulse Wk 33: The Competitor Got a PDUFA Date Five Days Later
Five days after Tarsus paid $450 million for a Stargardt asset reading out in 2029, the competitor got a PDUFA date of 12 February 2027.
The Pulse
NeuroPulse Wk 33: Jazz Bet $820M on a New FDA Evidence Standard
Jazz committed $820 million at signing for an epilepsy drug with one early trial behind it. The justification is a regulatory pathway, not the data.
OrphanPulse Wk 32: Tarsus Paid $450M Upfront for a Readout in 2029
Tarsus committed $450 million at signing for a Stargardt asset whose pivotal trial reads out in 2029, while a competitor is already filing.
NeuroPulse Wk 32: Takeda Fixed the Cause, and Cannot Ship It Yet
FDA approved the first medicine treating narcolepsy type 1 as a disease rather than a symptom set. Takeda cannot sell it for up to ninety days.
OrphanPulse Wk 31: The Panel Voted on a Process, Not a Drug
FDA advisers voted 9 to 3 against Capricor’s deramiocel, three weeks before PDUFA. The argument was not about biology. It was about which SAP counted.
NeuroPulse Wk 31: An Open-Label 77% Could Not Rescue a Missed Endpoint
FDA told NeuroPace its generalized epilepsy expansion is not approvable as filed. An open-label 77% cannot rescue a randomized endpoint that missed.
NeuroPulse Wk 30: Three Orexin Programmes in Two Weeks
Lundbeck’s Fast Track makes three orexin programmes in two weeks. Plus a Duchenne filing with a January date, and a CHMP slip caused by a contract manufacturer.
OrphanPulse Wk 30: CHMP Backs a Drug It Refused Twice
Nezglyal was refused in February 2024 and again in May. This week the same committee recommended it, and children with cerebral ALD get an oral option.
OrphanPulse Wk 29: Ionis Takes SMA-Proven Antisense Into Dravet Syndrome
A week defined by first-in-human starts across rare genetic disease, led by antisense entering Dravet syndrome from the field that already rewrote spinal muscular atrophy. Around it, first patients dosed in rare vascular and cardiomyopathy programs with near-empty competitive fields, a first-in-class readout in heparin-induced thrombocytopenia, and orphan designations that quietly reshape the economics behind them.
NeuroPulse Wk 29: First Brain Link Shares Movement Between Two People
The Alzheimer’s Association International Conference drove the week’s pipeline news, but the signal that mattered most came from a lab: the first brain link that let one paralysed person move and feel through another. Around it, a tau target validated and contested in the same week, an oral entrant scaling in Huntington’s, and anti-amyloid therapy fighting its real battle, which is access.